Health Condition 1: E755- Other lipid storage disorders
Conditions
Interventions
Intervention1: Taliglucerase alfa: Therapeutic class:
Lysosomal glucocerebroside specific enzyme
Dosage form:
Lyophilized Powder for solution for injection
Composition:
Water Content 3.7000 %
Sponsors
Prof Ari Zimran Shaare Zedek Medical Center Jerusalem Israel
Eligibility
Inclusion criteria
Inclusion criteria: 1.Male or female of any age; however, if female: must be using contraception if of childbearing potential or must be surgically sterile must not be lactating 2.Diagnosis of Type 3 GD by enzyme and sequence analysis; and confirmed by the Medical Monitor. 3.Splenomegaly at least 5 x multiples of normal (MN). 4.Treatment-naïve.
Exclusion criteria
Exclusion criteria: Any of the following is regarded as a criterion for exclusion from the trial: 1. Type 2 GD. 2. Presence of myoclonic seizures. 3. At least one allele of: • N370S (N409S in recent nomenclature) • R496H (R535H in recent nomenclature) 4. Presence of calcification in heart valves or arteries in echocardiography. 5. Presence of untreated iron, folic acid, vitamin B12 deficiency and/or hypothyroidism. (Resolved anemia is not an exclusion criterion.) 6. Presence of human immunodeficiency virus (HIV), hepatitis B surface antigen (HBsAg), and/or hepatitis C virus (HCV) infections 7. Splenectomy and bone marrow transplantation. 8. Presence of any medical, emotional, behavioural, or psychological condition that in the judgment of the Investigator would interfere with the subject’s compliance with the requirements of the study. 9. Any other disorder that may interfere with the results of the efficacy endpoints. 10. Pregnancy or breastfeeding. 11. Currently taking another investigational drug for any condition or any therapeutic drug for Gaucher disease. 12. The subject and/or subject’s parent(s) or legal guardian(s) are unable to understand the nature, scope, and possible consequences of the study. 13. Any medical history of food/drugs allergy.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Primary efficacy variable is: Percent change in spleen volume (expressed in MN) from baseline to Month 12 Timepoint: Primary efficacy variable is: Percent change in spleen volume (expressed in MN) from baseline to Month 12 | — |
Secondary
| Measure | Time frame |
|---|---|
| Percent change in Chitotriosidase from baseline to Months 3, 6, 9, and 12Timepoint: 3, 6, 9, and 12 months;Percent change in hemoglobin from baseline to Months 3, 6, 9, and 12Timepoint: 3, 6, 9, and 12 months;Percent change in Lyso-Gb1 from baseline to Months 3, 6, 9, and 12Timepoint: 3, 6, 9, and 12 months;Percent change in platelet count from baseline to Months 3, 6, 9, and 12Timepoint: 3, 6, 9, and 12 months;Secondary efficacy variables are: Percent change in liver volume (expressed in MN) from baseline to Month 12 Timepoint: 12 months | — |
Countries
India, Israel, Turkey
Contacts
Public ContactDr Madhulika Kabra
Division of Genetics (Department of Paediatrics)
Outcome results
None listed